Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.
Tiny capsules packed with gene-editing tools offer alternative to viral delivery of gene therapy
Posted on: 10 September 2019, source: University of Wisconsin
New tools for editing genetic code offer hope for new treatments for inherited diseases, some cancers, and even stubborn viral infections. But the typical method for delivering gene therapies to specific tissues in the body can be complicated and may cause troubling side effects. Researchers at the University of Wisconsin–Madison have addressed many of those problems by packing a gene-editing payload into a tiny, customizable, synthetic nanocapsule. They described the delivery system and its cargo today (Sept. 9, 2019) in the journal Nature Nanotechnology.
New tools for editing genetic code offer hope for new treatments for inherited diseases, some cancers, and even stubborn viral infections. But the typical method for delivering gene therapies to specific tissues in the body can be complicated and may cause troubling side effects. Researchers at the University of Wisconsin–Madison have addressed many of those problems by packing a gene-editing payload into a tiny, customizable, synthetic nanocapsule. They described the delivery system and its cargo today (Sept. 9, 2019) in the journal Nature Nanotechnology.
Gene Therapy Developers Issue Principles for Human Genome Editing
Posted on: 28 August 2019, source: raps.org
The Alliance for Regenerative Medicine’s (ARM) Gene Editing Task Force on Tuesday released a set of principles for human genome editing endorsed by thirteen of its members who are involved in the development of gene therapies or gene-editing technology. While the principles endorse somatic cell gene editing and the development of regulatory standards for gene editing, the document asserts that it is too early to support any form of human germline gene editing due to unanswered ethical, legal and safety questions. “As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles,” said ARM CEO Janet Lambert.
The Alliance for Regenerative Medicine’s (ARM) Gene Editing Task Force on Tuesday released a set of principles for human genome editing endorsed by thirteen of its members who are involved in the development of gene therapies or gene-editing technology. While the principles endorse somatic cell gene editing and the development of regulatory standards for gene editing, the document asserts that it is too early to support any form of human germline gene editing due to unanswered ethical, legal and safety questions. “As with all breakthrough biotechnologies, we need to exercise caution and good stewardship in our research and development practices and ensure that work involving the genetic modification of cells takes place within the bioethical framework outlined in these principles,” said ARM CEO Janet Lambert.
Novartis ousted top scientists over manipulation of data for gene therapy
Posted on: 14 August 2019, source: statnews.com
Novartis dismissed the top two scientists at its gene therapy division shortly after CEO Vas Narasimhan learned of internal data falsification that has since snowballed into a damaging scandal, a person familiar with the situation said Wednesday. The company previously said it was “in the process of exiting” scientists who were responsible for the scandal but did not identify them. In a statement on Wednesday, Novartis (NVS) said that it had appointed a new chief scientific officer for AveXis and that other scientists, Brian and Allan Kaspar, “have not been not been involved in any operations at AveXis since early May 2019,” without elaborating. The person familiar with the situation, who spoke on condition of anonymity, confirmed that the departure of the Kaspars, who are brothers, was connected to the disclosure of data manipulation related to the gene therapy Zolgensma.
Novartis dismissed the top two scientists at its gene therapy division shortly after CEO Vas Narasimhan learned of internal data falsification that has since snowballed into a damaging scandal, a person familiar with the situation said Wednesday. The company previously said it was “in the process of exiting” scientists who were responsible for the scandal but did not identify them. In a statement on Wednesday, Novartis (NVS) said that it had appointed a new chief scientific officer for AveXis and that other scientists, Brian and Allan Kaspar, “have not been not been involved in any operations at AveXis since early May 2019,” without elaborating. The person familiar with the situation, who spoke on condition of anonymity, confirmed that the departure of the Kaspars, who are brothers, was connected to the disclosure of data manipulation related to the gene therapy Zolgensma.
Gene Therapy Making Waves for Lung Cancer Patients
Posted on: 23 July 2019, source: mesothelioma.com
The development of lung cancer treatment have come a long way within the last decade. The progression of gene therapy through clinical trials is contributing to millions of lives extended as well as more patients going into remission. The success gene therapy is playing towards these common cancers are making waves in the rare space as well. An uncommon cancer affecting the lungs- mesothelioma, will hopefully start to leverage these types of treatments in the near future.
The development of lung cancer treatment have come a long way within the last decade. The progression of gene therapy through clinical trials is contributing to millions of lives extended as well as more patients going into remission. The success gene therapy is playing towards these common cancers are making waves in the rare space as well. An uncommon cancer affecting the lungs- mesothelioma, will hopefully start to leverage these types of treatments in the near future.
The Prickly Debate on Germline Gene Therapy, and Moving It Forward
Posted on: 26 June 2019, source: SingularityHub
In 2016, a healthy baby boy came screaming into the world in a Mexican clinic. Harboring DNA from three parents, the baby had had his genes dramatically altered while still an embryo. Without the treatment, an inheritable neurological disorder would’ve killed him before the age of three. Two years later across the world in China, two girls, also as embryos, had their genes edited using CRISPR to make them (theoretically) resistant to HIV infection. The edits didn’t hit their intended target—instead, the girls may be more prone to infections, have altered brain functions, and even experience earlier deaths.
In 2016, a healthy baby boy came screaming into the world in a Mexican clinic. Harboring DNA from three parents, the baby had had his genes dramatically altered while still an embryo. Without the treatment, an inheritable neurological disorder would’ve killed him before the age of three. Two years later across the world in China, two girls, also as embryos, had their genes edited using CRISPR to make them (theoretically) resistant to HIV infection. The edits didn’t hit their intended target—instead, the girls may be more prone to infections, have altered brain functions, and even experience earlier deaths.
Avexis Receives FDA Approval For Zolgensma, First And Only Gene Therapy For Pediatric Patients With Spinal Muscular Atrophy
Posted on: 25 May 2019, source: Novartis
AveXis, a Novartis company, today announced the US Food and Drug Administration (FDA) has approved Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.
AveXis, a Novartis company, today announced the US Food and Drug Administration (FDA) has approved Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time intravenous (IV) infusion. Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis.
NIH Streamlines Gene Therapy Oversight and Charts a Course for Considering Emerging Technology
Posted on: 26 April 2019, source: NIH
In August 2018, the National Institutes of Health and the Food and Drug Administration (FDA) announced a joint proposal to streamline duplicative and burdensome oversight over gene therapy. As part of this effort and following public input, NIH today released the amended NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules. The amendments remove the requirement to register and report on human gene therapy protocols under the NIH Guidelines to the NIH. Robust oversight continues under the FDA, which has regulatory oversight of all human gene therapy clinical trials. In addition, NIH-funded human gene therapy research remains subject to the usual NIH oversight that applies to all NIH-funded research, and oversight by local authorities such as Institutional Review Boards and Institutional Biosafety Committees.
In August 2018, the National Institutes of Health and the Food and Drug Administration (FDA) announced a joint proposal to streamline duplicative and burdensome oversight over gene therapy. As part of this effort and following public input, NIH today released the amended NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules. The amendments remove the requirement to register and report on human gene therapy protocols under the NIH Guidelines to the NIH. Robust oversight continues under the FDA, which has regulatory oversight of all human gene therapy clinical trials. In addition, NIH-funded human gene therapy research remains subject to the usual NIH oversight that applies to all NIH-funded research, and oversight by local authorities such as Institutional Review Boards and Institutional Biosafety Committees.
Gene Therapy News
- Alabama Man Free of Sickle Cell After Gene Therapy - WebMD
Tue, 17 Sep 2019 14:50 GMT - Reprogramming the Human Computer: Silicon Valley Meets Cell and Gene Therapy - BioBuzz
Tue, 17 Sep 2019 23:35 GMT - FDA approves 2nd gene therapy cancer drug from Durham's Precision Bio for clinical trial - WRAL Tech Wire
Tue, 17 Sep 2019 09:49 GMT - This Company Is Looking to Win Big in Gene Therapy - The Motley Fool
Tue, 17 Sep 2019 12:00 GMT - Advancements Made for Glaucoma Gene Therapy - Pharmacy Times
Wed, 24 Jan 2018 08:00 GMT
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Recent early view article in Journal of Gene Medicine:
You can buy a unique research paper online on gene therapy topics here.